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Oligonucleotides are short sequences of nucleotides (RNA or DNA), typically with twenty or fewer bases. Automated synthesizers allow the synthesis of oligonucleotides up to 160 to 200 bases.
CSHL researcher-inventor hails FDA approval of new SMA drug

CSHL researcher-inventor hails FDA approval of new SMA drug

Within a week of Christmas day, a drug called nusinersen will be in the hands of doctors across the nation, who will use it, most urgently, to treat young children with a severe and potentially fatal illness called spinal muscular atrophy (SMA). [More]
Ionis announces FDA approval of first SMA drug in the U.S for pediatric and adult patients

Ionis announces FDA approval of first SMA drug in the U.S for pediatric and adult patients

Ionis Pharmaceuticals, Inc. announced today that the U.S. Food and Drug Administration has approved SPINRAZATM (nusinersen) under Priority Review for the treatment of spinal muscular atrophy (SMA) in pediatric and adult patients. [More]
Turning off 'fat-specific protein 27' improves blood sugar levels, reduces body fat in animal model

Turning off 'fat-specific protein 27' improves blood sugar levels, reduces body fat in animal model

In a study published in the Journal of Lipid Research, Saint Louis University scientist Angel Baldan, Ph.D., reports that turning off a protein found in liver and adipose tissue significantly improves blood sugar levels, as well as reduces body fat in an animal model. [More]
Study offers promise for therapeutic management of fetal and congenital genetic diseases in utero

Study offers promise for therapeutic management of fetal and congenital genetic diseases in utero

A breakthrough study by research teams at Rosalind Franklin University of Medicine and Science and Oregon Health & Science University offers promise for therapeutic management of congenital diseases in utero using designer nucleotide sequences that can simply be injected into the fluid surrounding the developing fetus to potentially treat disabling-to-lethal genetic defects. [More]
Scientists find way to boost efficiency with which CRISPR-Cas9 cuts and disables genes

Scientists find way to boost efficiency with which CRISPR-Cas9 cuts and disables genes

CRISPR-Cas9 is the go-to technique for knocking out genes in human cell lines to discover what the genes do, but the efficiency with which it disables genes can vary immensely. [More]
Overcoming gene silencing barriers to target neurological conditions: an interview with Dr Errol de Souza

Overcoming gene silencing barriers to target neurological conditions: an interview with Dr Errol de Souza

Many gene mutations that cause neurological disorders have been identified. For example, in a rare neurological disorder such as Huntington’s disease, an autosomal dominant mutation through expansion of CAG (cytosine-adenine-guanine) triplet repeats in the gene coding for the Huntingtin protein results in abnormal protein production. [More]
Novel conjugate therapy shows promise in destroying ALL cells with few side effects

Novel conjugate therapy shows promise in destroying ALL cells with few side effects

Researchers at UC Davis and Ionis Pharmaceuticals have developed a hybrid treatment that harnesses a monoclonal antibody to deliver antisense DNA to acute lymphoblastic leukemia (ALL) cells and that may lead to less toxic treatments for the disease. [More]
Promising new therapeutic target could lead to better prognosis of spinal muscular atrophy

Promising new therapeutic target could lead to better prognosis of spinal muscular atrophy

According to studies, approximately one out of every 40 individuals in the United States is a carrier of the gene responsible for spinal muscular atrophy (SMA), a neurodegenerative disease that causes muscles to weaken over time. [More]
Duke to lead early-stage clinical trials for promising new neurological therapies

Duke to lead early-stage clinical trials for promising new neurological therapies

Duke University could receive up to $19 million to lead early-stage clinical trials for new drugs to treat neurological conditions such as Alzheimer's disease and neuropathy. [More]
Researchers discover novel neuroprotection strategies to slow progression of Parkinson's disease

Researchers discover novel neuroprotection strategies to slow progression of Parkinson's disease

Using a robust model for Parkinson's disease, University of Alabama at Birmingham researchers and colleagues have discovered an interaction in neurons that contributes to Parkinson's disease, and they have shown that drugs now under development may block the process. [More]
Automated liquid handling in pharmaceutical research: an interview with Dr Alfred Chappell

Automated liquid handling in pharmaceutical research: an interview with Dr Alfred Chappell

In developing antisense therapeutics, scientists at Ionis evaluate thousands of oligonucleotides. One of my responsibilities is to oversee the QC and management of oligonucleotides used in target screening. [More]
Researchers develop novel ss-siRNAs to turn off disease-related genes

Researchers develop novel ss-siRNAs to turn off disease-related genes

Combining the therapeutic potential and advantages of existing oligonucleotide-based approaches to turn off disease-related genes, a type of single-stranded silencing RNAs (ss-siRNAs) has shown significantly improved potency and activity. [More]
New, comprehensive UHPLC-MS workflow solutions provide in-depth biopharmaceutical characterization

New, comprehensive UHPLC-MS workflow solutions provide in-depth biopharmaceutical characterization

Biopharmaceutical companies can now achieve advanced levels of specificity, sensitivity and speed with specialized analytical workflow solutions for the characterization and quantification of proteins. [More]
Takara Bio USA, Integrated DNA Technologies collaborate to enhance transcriptome research

Takara Bio USA, Integrated DNA Technologies collaborate to enhance transcriptome research

Takara Bio USA, Inc. (TBUSA, formerly Clontech Laboratories, Inc.), a wholly owned subsidiary of Takara Bio Inc., and Integrated DNA Technologies (IDT) today announced a new collaboration to support targeted RNA sequencing. [More]
Study describes precise mechanisms that enable TB bacteria to persist in the body

Study describes precise mechanisms that enable TB bacteria to persist in the body

Bacteria that cause tuberculosis trick immune cells meant to destroy them into hiding and feeding them instead. This is the result of a study led by researchers from NYU Langone Medical Center and published online April 18 in Nature Immunology. [More]
SCIEX and QPS Holdings enter collaboration to establish the BioBA solution for bioanalysis of biologics

SCIEX and QPS Holdings enter collaboration to establish the BioBA solution for bioanalysis of biologics

SCIEX, a global leader in life science analytical technologies, and QPS Holdings, LLC (QPS), a leading global full-service Contract Research Organization (CRO), today announced a collaboration to advance Hybrid LBA –LCMS, a workflow for routine biologics quantification. Under the agreement, QPS Holdings will deploy the SCIEX BioBA Solution, the first end-to-end and most complete solution for bioanalysis of biologics. [More]
Novel plasmid-based microRNA inhibitor system effectively prevents microRNA activity in cells

Novel plasmid-based microRNA inhibitor system effectively prevents microRNA activity in cells

Today at the 45th Annual Meeting & Exhibition of the American Association for Dental Research, researcher Brad Amendt, University of Iowa, Iowa City, USA, will present a study titled "New Biotechnology to Inhibit MicroRNA Activity and Novel Applications for Craniofacial and Dental Research." [More]
Delivering microRNAs in cancer treatment: an interview with Dr Conde and Prof Artzi

Delivering microRNAs in cancer treatment: an interview with Dr Conde and Prof Artzi

microRNAs (miRs) are small endogenous noncoding RNA molecules (20–23 nucleotides) derived from imperfectly paired hairpin RNA structures naturally encoded in the genome that act specifically as triggering molecules to control translational repression or mRNA degradation. [More]
Using single-molecule studies to understand cellular processes: an interview with Professor W. E. Moerner

Using single-molecule studies to understand cellular processes: an interview with Professor W. E. Moerner

Single fluorescent molecules provide a local nanometer-sized probe of complex systems. We can measure the motion of the single molecule, use them to achieve imaging on a scale down to 20 nanometers, or we can infer aspects of the behaviour of the object under study by the details of the light that is emitted. [More]
Study paves way for development of novel cancer therapeutics

Study paves way for development of novel cancer therapeutics

A team of scientists, comprising researchers from the Institute of Molecular and Cell Biology, a research institute under the Agency for Science, Technology and Research, Singapore, and the VIB Laboratory of Molecular Cancer Biology, has revealed the mechanism by which tumor cells elevate levels of MDM4, a protein that is highly expressed in cancer cells but not in normal adult tissues. [More]
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